Amicus Therapeutics is Doing Great Things for Rare Orphan Diseases

Amicus Therapeutics was created in February of 2002, and is currently located in Cranbury, New Jersey, a tiny town in central New Jersey known best for the popular pharmaceutical firm specializing in developing treatments for rare diseases – none other than Amicus Therapeutics, itself.

The pharmaceutical company first went public with its stock in 2007, featuring the NASDAQ ticker symbol FOLD. Although Amicus Therapeutics used to be funded by a number of venture capitalist groups, it’s now able to fund its own operations through recently-acquired revenue streams and exclusive manufacturing agreements with various pharmaceutical producers.

Amicus Therapeutics, despite just beginning to sell treatments in select countries, has impressive financials. In its most recent release of financial statements in June of 2017, its total assets were slightly more than $900 million. Earlier this year, in August of 2017, Galafold – holding a chemical name of migalastat – was approved for human use in Australia. Galafold, to which Amicus Therapeutics holds the sole patent to, is designed to treat Fabry disease, a rare disease characterized by extremity pain and discomfort, crowds of red spots in patients’ torso areas, bad vision and hearing, tinnitus, and a host of other negative, virtually-unbearable symptoms. Prior to Amicus Therapeutics’ creation of Galafold, Fabry disease had no known treatments (http://www.gcreport.com/amicus-therapeutics-announces-new-drug-application/).

In 2017 alone, the share price of Amicus Therapeutics has nearly tripled, up 178% as of less than three weeks ago on September 10th, 2017 (ReleaseFact). In the month preceding 9/10/2017, the shares of Amicus Therapeutics rose slightly less than 7%, meaning its’ perceived value in financial markets is maintaining its status as an attractive investment.

Amicus Therapeutics focuses on creating treatments for orphan diseases, those with so few affected patients that pharmaceutical companies generally aren’t interested in creating treatments, cures, or mitigating agents for them – until Amicus Therapeutics came along, that is. The diseases that Amicus Therapeutics specializes in are called lysosomal storage disorders, or those featuring lysosomes – those that break down molecules – that have faulty enzymes that fail to break down microscopic bits of matter.